Melflufen (INN melphalan flufenamide) is a first in class peptide-drug conjugate (PDC) that targets aminopeptidases and rapidly releases alkylating agents into tumor cells. Melflufen is rapidly taken up by myeloma cells due to its high lipophilicity and is immediately hydrolyzed by peptidases to release an entrapped hydrophilic alkylator payload. Melflufen is an investigational drug and is not approved for commercial use.
Each year, approximately three in every 10,000 people are diagnosed with multiple myeloma*. While multiple myeloma can affect people of any age, the average age of diagnosis is 70 years of age. As there is no cure for multiple myeloma, a key focus of treatment is extending and improving the quality of life for patients. Melflufen is being developed for the treatment of patients with relapsed and refractory multiple myeloma.
Melflufen is currently being evaluated in the fully enrolled pivotal phase 2-study called HORIZON (OP-106) and Oncopeptides has submitted a New Drug Application (NDA) for accelerated approval of melflufen with FDA.
The phase 3 clinical study (OCEAN, OP-103) is currently enrolling and has been approved under the US Food and Drug Administration’s Special Protocol Assessment. In the OCEAN study, melflufen is compared directly against the current standard of care in patients with relapsed or refractory multiple myeloma.
As a rare condition, multiple myeloma is classified as an orphan disease in the US and Europe and melflufen has been granted orphan drug designation by the relevant authorities in both these jurisdictions.
* National Cancer Institute, SEER Cancer Statistics Review 1975-2013 (2016) for patients in USA and GlobalData (2015).